A Double-Blind, Placebo-Controlled, Crossover Study of Sacrosidase for the Treatment of Subjects With Fructan Intolerance
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- Change in incidence and severity of gastrointestinal symptoms in subjects with fructan intolerance treated with sacrosidase and placebo
研究概览
简要总结
SSDXA-14 is a Phase 2 (feasibility/pilot) double-blind, placebo-controlled, crossover study to evaluate the efficacy and safety of sacrosidase and placebo in 25 subjects objectively diagnosed with fructan intolerance.
详细描述
This Phase 2 study will evaluate the efficacy and safety of sacrosidase and placebo in male and female subjects aged 18 years or older objectively diagnosed with fructan intolerance via fructan breath test within 6 months prior to informed consent and are negative for inflammatory bowel disease or low-grade GI bacterial infection, detected by fecal calprotectin test, celiac disease, detected by serology for anti-gluten protein antibodies, congenital sucrase-isomaltase deficiency (CSID), detected by sucrose hydrogen-methane breath test, and have a negative result for abnormal uncontrolled thyroid function, detected by the standard TSH blood test. This study will consist of a Screening Visit, Baseline Period, Treatment Period 1, Washout Period, and Treatment Period 2. Subjects will be required to attend 5 clinic visits during study participation. Subjects will be randomized in a 1:1 fashion to either receive sacrosidase or placebo during Treatment Period 1. Following a 7-day Washout Period, subjects will receive their crossover study medication for Treatment Period 2. Subjects will take their assigned study medication for 7 days during each Treatment Period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to comprehend and provide a signed and dated informed consent form
- •Stated willingness to comply with all study procedures and attend all scheduled clinic visits, and continue participation for the duration of the study
- •Ability to self-administer oral medication and willingness to adhere to the medication regimen
- •Male or non-pregnant, non-lactating female, at least 18 years of age
- •Sexually active women of childbearing potential must agree to use at least one reliable method of birth control while participating in the study
- •Presence of fructan intolerance as determined by a positive result on a FBT within the last 6 months
- •Subjects who are lactose intolerant agree to eliminate all lactose from their diet during the study
- •Stated willingness to discontinue any medications to resolve GI symptoms (digestive enzymes, antacids, proton pump inhibitors, histamine-2 blockers, promotility agents, or anti-diarrheal agents, etc.), per the investigator's discretion.
- •Stated willingness to discontinue any over the counter or prescribed oral nonsteroidal anti-inflammatory drugs (NSAIDs) during the study
- •Per the discretion of the investigator, absence of any GI disorder other than a diagnosis of fructan intolerance
排除标准
- •History of inflammatory bowel disease or active low-grade GI bacterial infection, as diagnosed by the presence of fecal calprotectin
- •History of celiac disease, as diagnosed by serology testing for anti-gluten protein antibodies
- •History of CSID, as diagnosed by the sucrose hydrogen-methane breath test,
- •Abnormal uncontrolled thyroid function, detected by abnormal TSH level in the blood
- •Per the discretion of the investigator, history of a serious physical or mental disorder
- •BMI greater than 30 kg/m2
- •History of diabetes
- •History of hypersensitivity to yeast, yeast products, glycerin (glycerol), or papain
研究组 & 干预措施
Treatment
Subjects will be randomized to take active treatment, sacrosidase, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature) during either Treatment Period 1 or Treatment Period 2. The study treatment period is one week.
干预措施: Sacrosidase Oral Solution (Drug)
Placebo
Subjects will be randomized to take placebo treatment, sacrosidase placebo, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature) during either Treatment Period 1 or Treatment Period 2. The study treatment period is one week.
干预措施: Placebo (Drug)
结局指标
主要结局
Change in incidence and severity of gastrointestinal symptoms in subjects with fructan intolerance treated with sacrosidase and placebo
时间窗: Up to 7 weeks
Change from baseline as determined by the Daily Symptom Questionnaire Total Symptom Score (TSS) rating severity on a scale from 0 to 9 and frequency on a scale from 0 to 5, based on a mean value of combination scores for, incidence of seven GI symptoms experienced in the past 24 hours during a 7-day period and severity of seven GI symptoms experienced in the past 24 hours during a 7-day period.
Change From Baseline in Mean (SD) Total Symptom Score (TSS) at 1 Week
时间窗: Baseline and 1 week
The numeric value of the Total Symptom Score (TSS) is derived from the Daily Symptom Questionnaire (DSQ). The DSQ consisted of rating the frequency in the last 24 hours and the severity of 7 symptoms, abdominal bloating, abdominal pain, abdominal cramps, abdominal gas/flatulence, constipation, nausea, and diarrhea. The daily symptom score is calculated by summing the frequency and severity scores for each symptom with a minimum daily score of 0 and a maximum daily score of 98. The TSS is calculated by averaging the combined daily symptom scores during a 7-day period for baseline and 1-week post-intervention during each treatment period and ranges from 0-98, with higher values representing a worse outcome.
次要结局
- Change in incidence and severity of abdominal pain in subjects with fructan intolerance treated with sacrosidase and placebo(Up to 7 weeks)
- Assess the safety and tolerability of sacrosidase compared to placebo in subjects with fructan intolerance(Up to 1 year)
- Change From Baseline in Mean (SD) Frequency Score for Abdominal Bloating From Baseline to 1 Week(Baseline and 1 week)